Current Clinical Trials and Their Findings
Over 50 clinical trials using CRISPR or related gene-editing technologies are currently registered globally. Beyond blood disorders, researchers are testing treatments for inherited blindness, certain cancers, HIV, and rare genetic diseases. Each trial adds to our understanding of how to deploy these tools safely and effectively in humans.
A landmark 2024 study published in the New England Journal of Medicine reported that CRISPR-based editing of the TTR gene reduced a protein that causes hereditary transthyretin amyloidosis by 87% after a single infusion. Patients with this progressive heart and nerve disease had no other curative options. Six months after treatment, benefits persisted.
Cancer immunotherapy is another frontier. Researchers are editing T-cells to better recognize and destroy tumors, creating what are called CAR-T cells with enhanced persistence and reduced exhaustion. Early trials in leukemia and multiple myeloma patients have shown complete remissions in cases that failed all other therapies.
